Paroxysmal Nocturnal Hemoglobinuria (PNH), Persistent Anemia
Conditions
Keywords
C5 inhibitor, Complement Factor B (CFB) protein, Red Blood Cells
Brief summary
This study is researching an experimental drug called ALN-CFB. The study is focused on people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are currently taking a complement component C5 inhibitor ("C5-inhibitor") and continue to have anemia (low red blood cell count). The aim of the study is to see how tolerable ALN-CFB is compared to placebo. A placebo looks like the study drug but does not contain any drug. The study is looking at several other research questions, including: * What side effects may happen from taking ALN-CFB * How much ALN-CFB is in the blood at different times * How much Complement Factor B (CFB) protein levels in the blood are affected by ALN-CFB
Detailed description
The protocol will be amended to describe Part B of the study after Part A data have been analyzed.
Interventions
DRUGALN-CFB
Administered as defined in the protocol
DRUGPlacebo
Administered as defined in the protocol
Sponsors
Regeneron Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: 1. Has been diagnosed with PNH confirmed by a history of high flow cytometry from prior testing 2. Treated with a stable dose of C5 inhibitor (eculizumab or approved eculizumab biosimilar, ravulizumab, or crovalimab) for at least 24 weeks prior to screening visit, as described in the protocol 3. Has hemoglobin ≤10.5 g/dL at screening visit 1, with evidence of anemia prior to this visit, as described in the protocol 4. Has peripheral blood reticulocyte count of ≥100 x 10\^9/L at screening visit 1 Key
Exclusion criteria
1. Has history of bone marrow transplantation or receipt of an organ transplant 2. Has history of meningococcal infection or similar recurrent infections by other encapsulated bacterial organisms 3. Has any active, ongoing infection or a recent infection requiring ongoing systemic treatment with antibiotics, antivirals, or antifungals within 2 weeks of screening or during the screening period 4. Has laboratory evidence of bone marrow failure, as described in the protocol 5. Have recent, unstable medical conditions, not related to PNH or PNH-related complications, as described in the protocol NOTE: Other Protocol Defined Inclusion /
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Occurrence of Treatment-Emergent Adverse Events (TEAEs) | Through 365 Days |
| Severity of TEAEs | Through 365 Days |
Secondary
| Measure | Time frame |
|---|---|
| Concentrations of combined ALN-CFB and major metabolites in plasma | Through 365 Days |
| Concentrations of combined ALN-CFB and major metabolites in urine | Through 24 Hours following ALN-CFB administration |
| Absolute change from baseline in CFB concentration | Baseline, Through 365 Days |
| Percentage change from baseline in CFB concentration | Baseline, Through 365 Days |
Countries
Canada, South Korea, United Kingdom
Contacts
CONTACTClinical Trials Administrator
STUDY_DIRECTORClinical Trial Management
Regeneron Pharmaceuticals
Outcome results
None listed